Early treatment may help babies with SMA avoid breathing problems
NCT ID NCT02386553
First seen Nov 01, 2025 · Last updated May 02, 2026 · Updated 29 times
Summary
This study tested a drug called nusinersen in 25 infants who have the genetic markers for spinal muscular atrophy (SMA) but do not yet have symptoms. The goal was to see if giving the drug early could prevent or delay the need for breathing support or death. The drug is given via a spinal injection, and the study followed the infants over time to track their health outcomes.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611-2605, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Columbia University
New York, New York, 10032, United States
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David Geffen School of Medicine
Los Angeles, California, 90095, United States
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Fondazione Serena Onlus - Centro Clinico Nemo
Milan, 20162, Italy
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Hacettepe University Medical Faculty
Ankara, 6230, Turkey (Türkiye)
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Hamad General Hospital
Doha, 3050, Qatar
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Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, 807, Taiwan
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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National Taiwan University Hospital
Taipei, 100, Taiwan
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Nemours Children's Hospital, Orlando
Orlando, Florida, 32827, United States
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Ospedale Pediatrico Bambino Gesù
Rome, Lazio, 165, Italy
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Queensland Children's Hospital
South Brisbane, Queensland, 4101, Australia
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Royal Children's Hospital
Parkville, Victoria, 3052, Australia
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Seattle Children's Research Institute
Seattle, Washington, 98101, United States
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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The Johns Hopkins Hospital
Baltimore, Maryland, 21205, United States
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Universitaetsklinikum Freiburg
Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany
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University of California Davis Health System
Sacramento, California, 95817, United States
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University of Utah
Salt Lake City, Utah, 84112, United States
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Yeditepe University Medical School Hospital
Istanbul, 31755, Turkey (Türkiye)
Conditions
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